Andelyn Biosciences, a cell and gene therapy CDMO, has been selected to manufacture clinical grade AAV9-CSA vectors for UMass Chan Medical School.
These AAVs will be used to treat Cockayne syndrome, a rare genetic neurodegenerative disease categorised by impaired growth and development of the nervous system, photosensitivity and premature ageing.
The nonprofit patient advocacy organisation Riaan Research Initiative has made a sizeable donation to develop treatments for cockayne syndrome, and has partnered with Andelyn to make this happen.
This funding will allow the company to begin clinical trials for the disorder in the future, and Andelyn’s experience in gene therapy development and manufacturing will be leveraged to industrialise the production process and drive the programme’s success.
Miguel Sena-Esteves, Associate Professor of Neurology and Director of the Translational Institute for Molecular Therapeutics said: “Our dream of making a difference for Cockayne patients is coming closer every day, and signing the contract with Andelyn Biosciences to make the clinical material is a major step in that direction. Developing a gene therapy for fatal paediatric neurological diseases is challenging in many ways, but together with Riaan Research Initiative we have an outstanding team to bring our program to a first-in-human gene therapy clinical trial for Cockayne syndrome”.
Matt Niloff, Chief Commercial Officer of Andelyn, adds: “We are honored to have been selected to be a key contributor in the advancement of this critical therapy for Cockayne syndrome. With an increasing number of organisations across the industry adopting our data-driven and configurable AAV Curator Platform, we are excited to add UMass as an important partner leveraging the platform to drive the acceleration of the cockayne syndrome programme.”
